Randomized Controlled Study of Donepezil in Fragile X Syndrome
Status:
Completed
Trial end date:
2013-12-01
Target enrollment:
Participant gender:
Summary
Fragile X syndrome (FraX) is the most common known heritable cause of human intellectual
disability. Though recent research has revealed much about the genetic and neurobiological
bases of FraX, knowledge about specific and effective treatments for affected individuals is
lacking. Based on information from both human and animal studies, one cause of intellectual
disability in FraX may be related to deficits in a particular brain neurotransmitter system
(the "cholinergic" system). Thus, the investigators propose to use a specific medication,
donepezil, to augment cholinergic system in adolescents affected by FraX. If found to be
effective, the knowledge generated by this research may also be relevant to other
developmental disorders that share common disease pathways with FraX.
Phase:
Phase 2
Details
Lead Sponsor:
Stanford University
Collaborators:
Autism Speaks National Institute of Mental Health (NIMH)